The limitations of elderly citizens could be changed forever if a unique drug pioneered by Ochsner Pediatric Endocrinologist Dr. Robert Gensure proves as successful on humans as it has been on mice.
Gensure, who diagnoses, treats and manages hormonal disorders in children, has been researching treatments for osteoporosis, a silent disease that makes bones prone to fracture and is a major public health threat for more than 28 million Americans.
During their research, Gensure and University of Arkansas colleague Joshua Sakon made an important discovery – a drug that increases bone density in mice by up to 30 percent, which would prevent the natural bone loss that occurs over time.
“Bone density rises and falls with age, but this drug can increase the bone density in mice by 10 to 15 percent more than the peak and we saw as much as a 30 percent bone density increase,” Gensure said. “This means that it prevents the loss of bone that happens with age and the list of things elderly people can’t do would shrink.”
Gensure is originally from Boston, and it was there that he first began to take part in a study to treat osteoporosis. That study used parathyroid hormone, which is four to five times more effective than bisphosphonates – the most popular current treatment. The only problem with parathyroid hormone is that it has to be administered with daily injections.
“And that’s hard for a lot of people to keep up with,” Gensure said.
During the Boston study, the group was trying to find another delivery system for the hormone, such as a nasal spray. However, none of the other delivery systems panned out.
When Gensure came to Tulane and began working for Ochsner he met Sakon, who had a new collagen-binding delivery system.
“I told him I had something it could deliver,” Gensure said with a laugh. “We had a 50/50 shot of it working, and it did.”
The two began using weekly injections into mice that combined Sakon’s delivery system with the parathyroid hormone. The results were immediate.
“After eight weeks, the bone density in the mice had increased by 10 to 15 percent,” Gensure said.
According to Gensure, the most popular osteoporosis treatment currently on the market increases bone density by only 3 percent a year.
Eventually, it was discovered that weekly injections were too often and that only giving the mice the shots once was the better option.
“We gave the mice the drug used in chemo, which is the best at killing bone,” Gensure said. “This drug reversed that effect.”
Since mice have short age spans, Gensure is anxious to begin testing the drug on humans.
“We are in the process of getting permission from the FDA to begin human testing,” he said. “We need $500,000 to make the drug to begin human testing, and that’s the hard part.”
Gensure said that his funding opportunities have increased since President Obama took office and he has already applied for three grants this year. He is also working with several interested companies.
“Our goal is to get the drug out there, but a lot of it is in the hands of the FDA,” Gensure said. “Ten years is what people typically quote when it comes to getting the drug to the public. If we don’t hit any snags, it could be shorter. If we do, it could be longer than that.”
But right now, Gensure is pushing to get the drug, which has no known side effects, into human testing.
“Something like this can do better than what’s currently available to treat osteoporosis and there is a need for something that does better,” he said. “If it works in humans like it has in mice, it would not only be used to treat osteoporosis, but could be given to the elderly and athletes to prevent the natural bone loss that occurs over time.”
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